Overview: A potential breakthrough in multiple sclerosis treatment
Multiple sclerosis (MS) is a chronic autoimmune disease where the body’s immune system damages the myelin sheath that insulates nerve fibers. This disruption slows or blocks nerve signals, leading to symptoms such as numbness, vision loss, and paralysis. While existing therapies help reduce inflammation, there are no approved treatments that reliably protect neurons or repair the myelin itself. Recent research offers a glimmer of hope: two new drug candidates, K102 and K110, show promise in promoting remyelination and modulating immune responses.
From discovery to potential therapy
The work, published in Scientific Reports, builds on a long-standing collaboration between researchers at the University of California, Riverside (UCR) and the University of Illinois Urbana-Champaign (UIUC). Led by Seema Tiwari-Woodruff and John Katzenellenbogen, the team identified two lead compounds—K102 and K110—among more than 60 analogs derived from a previously studied compound, indazole chloride. These candidates improved safety, efficacy, and drug-like properties in mouse models and human cells, addressing the key challenge of translating animal data to human patients.
K102: A lead candidate with dual potential
K102 emerged as the lead compound due to its ability to promote remyelination and modulate immune function, a balance researchers deem crucial for MS therapies. Notably, K102 demonstrated activity in human oligodendrocytes—the myelin-producing cells of the central nervous system—derived from induced pluripotent stem cells. This finding suggests the compound could bridge the gap between preclinical models and human disease, increasing the likelihood of successful clinical translation.
K110: A strong backup with broader potential
While K102 takes the lead, K110 also shows promise, with slightly different central nervous system effects that could make it suitable for other conditions like spinal cord injury or traumatic brain injury. Keeping both candidates in development reflects a strategy to cover a broader range of demyelinating and neuroinflammatory conditions, not just MS.
The path to clinical testing
The research program benefited from the National MS Society’s funding, including traditional investigator-initiated grants and the Fast Forward commercial accelerator. Fast Forward accelerates the transition from academic discovery to industry collaboration by supporting data generation and licensing activities. This funding enabled the UCR-UIUC team to license the rights to Cadenza Bio, which is now advancing K102 and K110 toward non-clinical studies and, eventually, first-in-human trials.
Industry and academic collaboration: turning science into real-world impact
Patents for the K102/K110 program are jointly held by UCR and UIUC, with exclusive worldwide licensing to Cadenza Bio. The arrangement illustrates how long-standing academic partnerships can yield tangible patient benefits through translational science. Leaders from UCR, UIUC, and Cadenza Bio emphasize that the goal is to move from slowing axon damage to repairing it, potentially reducing long-term disability for people living with MS.
Looking ahead
Beyond MS, the team envisions applying these insights to other neurological diseases characterized by demyelination or neuronal injury, including stroke and neurodegeneration. Cadenza Bio is conducting necessary non-clinical work to support first-in-human studies, with hopes that clinical trials could begin soon. While challenges remain, the K102 and K110 program represents a meaningful step toward a first-in-class therapy that restores myelin and improves nerve function in MS patients.
As researchers push this work toward patients, the broader MS community watches with cautious optimism, hopeful that remyelination therapies could one day complement anti-inflammatory drugs and change the disease trajectory for millions worldwide.
